The FDA granted priority review for the experimental, all-oral Gaucher's disease treatment.
The agency extended the review time for Biogen's Alprolix by three months.
The patient population is small, at around 3,000, but the expected price tag is $330,000 a year.
The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.
The US-only recall amounts to less than 1% of the company's inventory.
The regulator says the drugmaker needs to do more testing.
Eli Lilly is the first Big Pharma to team up with the agency in its rare-disease pre-clinical development program.
Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.
Takeda moves an Alzheimer's treatment to Phase III; Sangamo acquires Ceregene; Catalyst gets breakthrough designation for auto-immune disorder drug; study says seniors likely to opt out of health insurance
Australia's price watchdogs are assessing the value of BMS cancer drug Yervoy; why a Phase II weight drug from Zafgen could open up the category; GSK halts a Crohn's trial; and Shire extends Santaris rare-disease collaboration
The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.
Alexion plans on moving into new HQ in 2015, AMA declares obesity is a disease, FDA looks into Zyprexa patient deaths
Genzyme is taking rare disease awareness to another level with a documentary film, dubbed "Facing Fabry Together," that follows four families grappling with the genetic disorder.
A study shows FDA approvals and awareness efforts are having little impact on physician awareness or patient frustration.
Industry observances of Rare Disease Awareness Day are getting more elaborate of late—perhaps not solely for altruistic reasons, as a third of NMEs approved by FDA over the past five years were for the treatment of rare diseases.
Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.
A recent study says that the financial incentives go beyond taxes and grants and that investing in R&D can mean entry into a space that's grown 26% from 2001 to 2010.
PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.